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Rare Disease

Fabry film spotlights a rare disease

Matthew Arnold April 30, 2013

Genzyme is taking rare disease awareness to another level with a documentary film, dubbed "Facing Fabry Together," that follows four families grappling with the genetic disorder.

Rare disease awareness fails to ease physician, patient burden

Deborah Weinstein April 15, 2013

A study shows FDA approvals and awareness efforts are having little impact on physician awareness or patient frustration.

Rare disease awareness more than just a day for pharmas

Deborah Weinstein February 28, 2013

Industry observances of Rare Disease Awareness Day are getting more elaborate of late—perhaps not solely for altruistic reasons, as a third of NMEs approved by FDA over the past five years were for the treatment of rare diseases.

Drug for ultrarare lipid disorder OK'd by FDA

Marc Iskowitz December 26, 2012

Aegerion's Juxtapid is first to market for treating the ultrarare inherited cholesterol condition HoFH, or homozygous familial hypercholesterolemia.

Rare diseases worth the investment, study says

Deborah Weinstein August 22, 2012

A recent study says that the financial incentives go beyond taxes and grants and that investing in R&D can mean entry into a space that's grown 26% from 2001 to 2010.

Company news: Burson-Marsteller, Corcept and Orexigen

August 07, 2012

PR firm Burson-Marsteller launched a new healthcare subsidiary, Orexigen's net loss widened in Q2, and Corcept closed out its first full quarter with orphan treatment Korlym.

Web Exclusive

What NOT to say to an MS Patient

Dorothy Wetzel June 07, 2013

It is often hard to find the right words when talking to a patient, friend or family member with MS. Even with training and awareness of the disease, we often blurt out something we think is innocuous only to find out later that we have deeply offended. The Health Activists that Extrovertic spoke to had some suggestions for having positive conversations with someone with MS.

2013 Hall of Fame Inductees

An entertaining look at the extraordinary lives and careers of the Medical Advertising Hall of Fame's 2013 inductees: Peter Frishauf, Dorothy Philips and David Labson. Clips produced by JUICE Pharma.